small interfering RNA targeting complement component 4 binding protein alpha

Small interfering RNA targeting complement component 4 binding protein alpha is an experimental RNA interference (RNAi) therapeutic designed to silence the expression of the *C4BPA* gene. Research indicates that C4BPA is significantly upregulated in fibroadipogenic precursor cells (FAPs) within the muscles of patients with Duchenne muscular dystrophy (DMD). High levels of secreted C4BPA act as an anti-myogenic factor, inhibiting the differentiation of muscle progenitor cells and impairing contractile function. By utilizing siRNA to degrade C4BPA mRNA, researchers have demonstrated a partial restoration of myogenic capacity and improved nuclear content per myotube in DMD-derived cultures. This approach represents a novel strategy for modulating the fibrotic environment and preserving muscle regeneration in dystrophic muscles.

Other names

C4BPA siRNAC-4BPA siRNAC 4BPA siRNAsiRNA targeting C4BPA